d367504f-f25e-44ad-ae5d-bd08d5dd5511.pdf ABLYNX TO PRESENT A POST-HOC ANALYSIS OF THE PHASE II TITAN STUDY WITH CAPLACIZUMAB IN ACQUIRED TTP PATIENTS AT THE 21st CONGRESS OF THE EUROPEAN HEMATOLOGY ASSOCIATION Post-hoc analysis of the TITAN data showed that treatment with caplacizumab significantly reduced a composite endpoint of major thromboembolic events and mortality

GHENT, Belgium, 26 May 2016 - Ablynx [Euronext Brussels: ABLX; OTC: ABYLY] today announced that a post- hoc analysis of the worldwide Phase II TITAN study of its wholly-owned Nanobody®, caplacizumab, in patients with acquired thrombotic thrombocytopenic purpura (aTTP), will be presented at the 21st Congress of the European Hematology Association (EHA), being held from 9-12 June 2016 in Copenhagen, Denmark.

It has been previously reported that the efficacy and safety of caplacizumab in conjunction with the standard of care (plasma exchange) were evaluated in a Phase II study in 75 patients with aTTP. Caplacizumab was well- tolerated and the primary endpoint of a reduction in time to platelet normalisation was achieved with statistical significance (p= 0.005). In addition, during treatment, caplacizumab reduced aTTP recurrences by 71% compared to placebo when administered as an adjunct to standard of care1.

Despite the current standard of care, mortality from an episode of acquired TTP is still reported to be up to 20% and patients remain at risk of life-threatening thrombotic complications. A post-hoc analysis of the Phase II data was performed to assess the impact of caplacizumab on a composite endpoint of major thromboembolic events and TTP-related mortality. The results demonstrate that a significantly lower proportion of subjects treated with caplacizumab experienced one or more major thromboembolic events, or died, as compared to placebo (11.4% versus 43.2%, nominal p-value of 0.006). These clinically meaningful results suggest that treatment with caplacizumab has the potential to reduce the major morbidity and mortality associated with acquired TTP.

The results from this post-hoc analysis will be presented during a poster presentation on 10 June 2016, from 17:15 to 18:45 CET (poster Hall H). The abstract (LB418): "Impact of caplacizumab treatment on mortality and major thromboembolic events in acquired TTP: Phase II TITAN study results", is available on the EHA website at www.ehaweb.org.

About caplacizumab and the TITAN study results

Caplacizumab is a highly potent and selective bivalent anti-von Willebrand Factor (vWF) Nanobody that received Orphan Drug Designation in the USA and EU in 2009. Caplacizumab inhibits the interaction between ultra-large vWF and platelets by targeting the A1 domain of vWF. It thereby prevents platelet aggregation and the formation of micro-clots during the acute, critical phase of acquired TTP.

Caplacizumab's clinical effect was demonstrated in the Phase II TITAN study in 75 patients with aTTP:

  • As indicated by a nearly 40% reduction in median time to platelet count normalisation (p = 0.005). Treatment with caplacizumab reduced the use of daily plasma exchange (PEX) and prevented further formation of micro clots and small blood vessel occlusion.

  • As shown by the low number of recurrences requiring re-initiation of daily plasma exchange during treatment with caplacizumab (N=3) vs. placebo (N=11).

1 Press release June 2014; Manuscript in the NEJM, Feb 2014

These results will serve as the basis for filing for conditional approval of caplacizumab in Europe in H1 2017. The confirmatory Phase III HERCULES study is currently ongoing to support the BLA filing in the USA. Results from this Phase III study are expected by the end of 2017. Caplacizumab could be the first therapeutic specifically approved for the treatment of acquired TTP.

About aTTP

aTTP is an acute life-threatening, ultra-rare, blood clotting disorder, affecting up to 11 per million people worldwide. It has a sudden onset caused by impaired activity of the ADAMTS13 enzyme (typically 2, resulting in thrombotic complications and widespread organ damage3.

aTTP is associated with major morbidities in the brain (e.g. stroke), heart and kidney and impacts life expectancy and quality of life4. Mortality is high at 10-20%5, typically occurring within 2 weeks after initial diagnosis. Moreover, about 36% of patients have recurrences6 after treatment with the current standard of care, which consists of plasma exchange and immune-suppressants, and these recurrences have the potential to cause further organ damage, thrombotic complications and poorer longer term outcomes.

About Ablynx

Ablynx is a biopharmaceutical company engaged in the development of Nanobodies®, proprietary therapeutic proteins based on single-domain antibody fragments, which combine the advantages of conventional antibody drugs with some of the features of small-molecule drugs. Ablynx is dedicated to creating new medicines which will make a real difference to society. Today, the Company has more than 40 proprietary and partnered programmes in development in various therapeutic areas including inflammation, haematology, immuno- oncology, oncology and respiratory disease. The Company has collaborations with multiple pharmaceutical companies including AbbVie, Boehringer Ingelheim, Eddingpharm, Genzyme, Merck & Co., Inc., Merck KGaA, Novartis, Novo Nordisk and Taisho Pharmaceuticals. The Company is headquartered in Ghent, Belgium. More information can be found on www.ablynx.com.

For more information, please contact:

Ablynx

Dr Edwin Moses CEO

t: +32 (0)9 262 00 07

m: +32 (0)473 39 50 68

e: edwin.moses@ablynx.com

Marieke Vermeersch

Associate Director Investor Relations t: +32 (0)9 262 00 82

m: +32 (0)479 49 06 03

e: marieke.vermeersch@ablynx.com@AblynxABLX

Ablynx media/analyst relations: FTI Consulting

Julia Phillips, Brett Pollard, Mo Noonan, Matthew Moss t: +44 20 3727 1000

e: ablynx@fticonsulting.com

2 Veyradier, NEJM 2016: "von Willebrand Factor - A new target for TTP treatment?"

3 Scully et al, Br J Hem 2012; Sarode et al, J Clin Apher 2014; Chaturvedi et al, Am J Hem 2013

4 Deford Blood 2013

5 Allford et al, BJH 2003, Kremer Hovinga, Blood 2010; Benhamou, Haematologica 2012; Goel et al Transfusion 2016

6 George et al, EJB 2008

Disclaimer

Certain statements, beliefs and opinions in this press release are forward-looking, which reflect the Company or, as appropriate, the Company directors' current expectations and projections about future events. By their nature, forward-looking statements involve a number of risks, uncertainties and assumptions that could cause actual results or events to differ materially from those expressed or implied by the forward-looking statements. These risks, uncertainties and assumptions could adversely affect the outcome and financial effects of the plans and events described herein. A multitude of factors including, but not limited to, changes in demand, competition and technology, can cause actual events, performance or results to differ significantly from any anticipated development. Forward looking statements contained in this press release regarding past trends or activities should not be taken as a representation that such trends or activities will continue in the future. As a result, the Company expressly disclaims any obligation or undertaking to release any update or revisions to any forward-looking statements in this press release as a result of any change in expectations or any change in events, conditions, assumptions or circumstances on which these forward-looking statements are based. Neither the Company nor its advisers or representatives nor any of its parent or subsidiary undertakings or any such person's officers or employees guarantees that the assumptions underlying such forward-looking statements are free from errors nor does either accept any responsibility for the future accuracy of the forward-looking statements contained in this press release or the actual occurrence of the forecasted developments. You should not place undue reliance on forward-looking statements, which speak only as of the date of this press release.

Ablynx NV published this content on 26 May 2016 and is solely responsible for the information contained herein.
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